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27
A PHASE 1/2 OPEN LABEL EXTENSION STUDY OF GIVOSIRAN, AN INVESTIGATIONAL RNAI THERAPEUTIC, IN PATIENTS WITH ACUTE INTERMITTENT PORPHYRIA
R. Marchia, L. Duartea, S. Pb, R. Db, A. Kec, B. Hld, S. Ee, H. Pe, B. Mf, E. Ala
a Alnylam Brasil Farmacêutica Ltda, São Paulo, SP, Brazil
b King's College Hospital, London, United Kingdom
c University of Texas Medical Branch, Galveston, United States
d Wake Forest University NC Baptist Medical Center, Winston-Salem, United States
e Porphyria Centre Sweden, Centre for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm, Sweden
f Icahn School of Medicine at Mt. Sinai, New York, United States
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ISSN: 25311379
Original language: English
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Hematology, Transfusion and Cell Therapy